New cellular entry pathway improves gene therapy outcomes

Alexia Bădoiu

Centenary Institute Jul 15 2025 Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy, Pompe disease and hemophilia. Published in the leading journal Cell , the study identifies a previously unknown gateway into human cells, a receptor called AAVR2, that gene therapy viruses use to deliver therapeutic genes. This

New cellular entry pathway improves gene therapy outcomes https://www.news-medical.net - 11:30

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New cellular entry pathway improves gene therapy outcomes https://www.news-medical.net - 11:30